OBJECTIVES: Congenital hyperinsulinism (CHI) is an ultra-rare disease impacting approximately 1:25,000–50,000 newborns. Unregulated insulin secretion causes hypoglycemia in infants, with potential consequences including seizures, brain damage, and death. Pharmacological interventions include continuous glucose infusions, diazoxide, somatostatins, and surgery. Diazoxide, the only approved treatment for hyperinsulinism-induced hypoglycemia, is ineffective in up to 60% of cases, necessitating the off-label use of other medications, including somatostatins. Surgical interventions pose risks, including potential long-term consequences, such as insulin-dependent diabetes. Dasiglucagon, an anti-hypoglycemic agent, is currently being investigated as a potential CHI treatment by Zealand Pharma. As no CHI-specific US economic analyses have been published, an economic model is needed to assess the costs associated with present and future CHI treatments.

METHODS: A 5-year budget impact model (BIM) was created from a US commercial perspective, assessing CHI newborns and children up to 18 years of age. A patient pathway was developed incorporating clinical guidelines and expert opinions, with dasiglucagon as either a second- or first-line treatment, using diazoxide as the default first-line option. Informed by a targeted literature review, the model incorporated clinical inputs, pharmacy, medical, surgical, and adverse event costs.

RESULTS: The BIM predicts that introducing dasiglucagon as a first- or second-line treatment decreases the need for additional medical or surgical interventions, minimizing the likelihood of enduring long-term consequences, like insulin-requiring diabetes, and their associated costs. Additionally, dasiglucagon's favorable safety profile reduces the occurrence of adverse events, thereby lowering the associated costs. The greatest cost reductions were observed when dasiglucagon was a first-line treatment.

CONCLUSIONS: The de-novo BIM includes clinical data, costs, and published literature, accommodating uncertainty through sensitivity and scenario analyses. While the BIM does not account for dasiglucagon’s improved glycemic control, dasiglucagon is anticipated to expedite hospital discharge and lower resource utilization. Future cost-effectiveness analyses should encompass these outcomes.